Marisa Wexler

Mutations in the ACVRL1 gene may play a role in the development of idiopathic pulmonary fibrosis (IPF), and targeting the gene — until now, little noted, according to researchers — could potentially lead to new treatments for the respiratory disease, a st... See more

Evrysdi shown to stabilize motor, lung function in adults with SMA: Study Gains were particularly notable in severely affected patients, researchers said Written by | - Evrysdi treatment for SMA stabilizes motor and lung function in adults over 3 years, a... See more

FDA denies accelerated approval of bitopertin, awaits trial results Positive data from ongoing Phase 3 trial could support traditional approval Written by | The U.S. Food and Drug Administration (FDA) has rejected an application from Disc Medicine seeking... See more

Hair metal patterns differ in people with Parkinson’s, new study suggests Lower iron was the most consistent change across people and mice Written by | - Parkinson’s patients show altered hair metal patterns, notably lower iron levels. - Mouse studies sug... See more

Early results of trial testing tebapivat in SCD expected later this year Enrollment now complete at 31 study sites in North America and Europe Written by | A small clinical trial testing Agios Pharmaceuticals’ oral therapy candidate tebapivat against a pl... See more